CL2019003420A1 - Compositions and methods for decreasing the expression of tau, useful for treating diseases associated with tau. (divisional request 201801652) - Google Patents

Compositions and methods for decreasing the expression of tau, useful for treating diseases associated with tau. (divisional request 201801652)

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Publication number
CL2019003420A1
CL2019003420A1 CL2019003420A CL2019003420A CL2019003420A1 CL 2019003420 A1 CL2019003420 A1 CL 2019003420A1 CL 2019003420 A CL2019003420 A CL 2019003420A CL 2019003420 A CL2019003420 A CL 2019003420A CL 2019003420 A1 CL2019003420 A1 CL 2019003420A1
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Chile
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tau
protein
compositions
methods
expression
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CL2019003420A
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Spanish (es)
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Ofengeim Manuela Polydoro
Jan Weiler
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Novartis Ag
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Publication of CL2019003420A1 publication Critical patent/CL2019003420A1/en

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Abstract

SE PROPORCIONAN EN ESTA SOLICITUD COMPOSICIONES Y MÉTODOS PARA DISMINUIR LA EXPRESIÓN DE PROTEÍNA Y ARNM TAU. ESTAS COMPOSICIONES Y MÉTODOS SON ÚTILES EN EL TRATAMIENTO DE ENFERMEDADES Y TRASTORNOS RELACIONADOS CON TAU. PARTICULARMENTE, SE PROPORCIONAN EN LA PRESENTE SOLICITUD OLIGONUCLEÓTIDOS ANTISENTIDO QUE SE DIRIGEN A LA PROTEÍNA TAU ASOCIADA A MICROTÚBULO HUMANA (MAPT, CONFORME A SUS SIGLAS EN INGLÉS), COMPOSICIONES QUE COMPRENDEN LOS OLIGONUCLEÓTIDOS ANTISENTIDO, Y MÉTODOS PARA DISMINUIR LA EXPRESIÓN DE PROTEÍNA Y ARNM DE TAU USANDO ESTOS OLIGONUCLEÓTIDOS ANTISENTIDO. LAS COMPOSICIONES Y LOS MÉTODOS PROPORCIONADOS EN ESTE DOCUMENTO SON ÚTILES EN EL TRATAMIENTO DE ENFERMEDADES ASOCIADAS A TAU.COMPOSITIONS AND METHODS TO DECREASE THE EXPRESSION OF PROTEIN AND TAU mRNA ARE PROVIDED IN THIS APPLICATION. THESE COMPOSITIONS AND METHODS ARE USEFUL IN THE TREATMENT OF TAU-RELATED DISEASES AND DISORDERS. PARTICULARLY, IN THIS APPLICATION, ANTI-SENSE OLIGONUCLEOTIDES ARE PROVIDED THAT TARGET THE TAU PROTEIN ASSOCIATED WITH THE HUMAN MICROTUBE (MAPT, ACCORDING TO ITS ACRONYMS IN ENGLISH), COMPOSITIONS THAT INCLUDE THE PROTEIN OF THE PROTEIN ASSOCIATED WITH THE HUMAN MICROTUBLE (MAPT, ACCORDING TO ITS ACRONYM IN ENGLISH), COMPOSITIONS THAT INCLUDE THE PROTEIN OF THE ANTI-PRINTED OLIGONUCLEOTIDE, AND THE EXPRESSED PROTEIN, AND THE EXPRESSED PROTEIN OF THE PROTEIN, AND THE EXPRESSION OF THE PROTEIN OF ANTI-PRINTED MISCELLANEOUS PROTEIN. TAU USING THESE ANTI-SENSE OLIGONUCLEOTIDES. THE COMPOSITIONS AND METHODS PROVIDED IN THIS DOCUMENT ARE USEFUL IN THE TREATMENT OF DISEASES ASSOCIATED WITH TAU.

CL2019003420A 2015-12-21 2019-11-25 Compositions and methods for decreasing the expression of tau, useful for treating diseases associated with tau. (divisional request 201801652) CL2019003420A1 (en)

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US201562270165P 2015-12-21 2015-12-21

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CL2018001652A CL2018001652A1 (en) 2015-12-21 2018-06-18 Compositions and methods to reduce tau expression
CL2019000149A CL2019000149A1 (en) 2015-12-21 2019-01-18 Compositions and methods to reduce the expression of tau, useful for treating diseases associated with tau. (divisional application 201801652)
CL2019003420A CL2019003420A1 (en) 2015-12-21 2019-11-25 Compositions and methods for decreasing the expression of tau, useful for treating diseases associated with tau. (divisional request 201801652)

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CL2018001652A CL2018001652A1 (en) 2015-12-21 2018-06-18 Compositions and methods to reduce tau expression
CL2019000149A CL2019000149A1 (en) 2015-12-21 2019-01-18 Compositions and methods to reduce the expression of tau, useful for treating diseases associated with tau. (divisional application 201801652)

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EP (1) EP3394259B1 (en)
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KR (1) KR20180089517A (en)
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Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
TW202246503A (en) 2013-07-19 2022-12-01 美商百健Ma公司 Compositions for modulating tau expression
JOP20190065A1 (en) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing tau expression
MX2020009532A (en) * 2018-03-13 2020-10-05 Janssen Pharmaceutica Nv Modified oligonucleotides for use in treatment of tauopathies.
KR20230143199A (en) 2018-07-03 2023-10-11 에프. 호프만-라 로슈 아게 Oligonucleotides for modulating tau expression
CN116234585A (en) * 2020-03-30 2023-06-06 阿尔尼拉姆医药品有限公司 Microtubule-associated protein TAU (MAPT) iRNA agent compositions and methods of use thereof
CN113817728A (en) * 2020-06-19 2021-12-21 四川大学华西医院 Recombinant lentivirus capable of effectively interfering Tau protein expression and application thereof
KR20240034185A (en) * 2021-06-16 2024-03-13 엠피리코 인크. Treatment of MTRES1-related diseases and disorders
WO2024091874A1 (en) * 2022-10-25 2024-05-02 Empirico Inc. Gene therapy treatment of mtres1 related diseases and disorders

Family Cites Families (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (en) 1970-11-10 1977-09-15 Organon Nv PROCEDURE FOR DETERMINING AND DETERMINING LOW MOLECULAR COMPOUNDS AND PROTEINS THAT CAN SPECIFICALLY BIND THESE COMPOUNDS AND TEST PACKAGING.
US3817837A (en) 1971-05-14 1974-06-18 Syva Corp Enzyme amplification assay
US3939350A (en) 1974-04-29 1976-02-17 Board Of Trustees Of The Leland Stanford Junior University Fluorescent immunoassay employing total reflection for activation
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4275149A (en) 1978-11-24 1981-06-23 Syva Company Macromolecular environment control in specific receptor assays
US4277437A (en) 1978-04-05 1981-07-07 Syva Company Kit for carrying out chemically induced fluorescence immunoassay
US4366241A (en) 1980-08-07 1982-12-28 Syva Company Concentrating zone method in heterogeneous immunoassays
AU651569B2 (en) 1990-01-11 1994-07-28 Isis Pharmaceuticals, Inc. Compositions and methods for detecting and modulating RNA activity and gene expression
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020052331A1 (en) 1990-08-14 2002-05-02 Brenda Baker Compositions and methods for antisense inhibition of protein translation
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
US7408027B1 (en) 1991-12-06 2008-08-05 Max-Planck-Gesellschaft Zur Forderung Der Wissenchaften Tools for the diagnosis and treatment of Alzheimer's disease
DK0618968T3 (en) 1991-12-06 2000-04-10 Max Planck Gesellschaft Means for diagnosing and treating Alzheimer's disease
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
CA2116460A1 (en) 1993-03-02 1994-09-03 Mitsubishi Chemical Corporation Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being
KR940021073A (en) 1993-03-02 1994-10-17 미야베 요시가즈 Prophylactic or therapeutic agents for Alzheimer's disease, methods for screening the same, and tau protein kinase I derived from humans
JPH08510130A (en) 1993-05-11 1996-10-29 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Antisense oligonucleotides that inhibit aberrant splicing and methods of using the same
BR9707529A (en) 1996-02-14 2000-01-04 Isis Pharmaceuticals Inc Oligunucleotide specifically hybridized with DNA or RNA.
JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
NZ503765A (en) 1997-09-12 2002-04-26 Exiqon As Bi-cyclic and tri-cyclic nucleotide analogues
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
CA2330944A1 (en) 1998-05-01 1999-11-11 Mayo Foundation For Medical Education And Research Pathogeniv tau mutations
US6664443B1 (en) 1998-05-01 2003-12-16 Mayo Foundation For Medical Education And Research Pathogenic Tau mutations in transgenic mice
CZ2001149A3 (en) * 1998-07-14 2002-02-13 Corixa Corporation Preparations and methods for therapy and diagnostic of prostate carcinoma
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
WO2000064262A1 (en) 1999-04-26 2000-11-02 The University Of North Carolina At Chapel Hill Antisense human fucosyltransferase sequences and methods of use thereof
CN101284873A (en) * 1999-06-30 2008-10-15 科里克萨有限公司 Compositions and methods for the therapy and diagnosis of lung cancer
WO2001053340A2 (en) 2000-01-21 2001-07-26 Pharmacia & Upjohn Company Transgenic mouse model of human neurodegenerative disease
CA2400595A1 (en) * 2000-02-28 2001-09-07 Decode Genetics Ehf Human schizophrenia gene
AU2002231741A1 (en) 2001-01-11 2002-07-24 Bayer Aktiengesellschaft Regulation of human tau-tubulin kinase
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
WO2004003134A2 (en) 2002-06-26 2004-01-08 Isis Pharmaceuticals, Inc. Antisense inhibition via rnase h-independent reduction in mrna
US20080176812A1 (en) 2002-08-05 2008-07-24 Davidson Beverly L Allele-specific silencing of disease genes
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
WO2004016655A1 (en) 2002-08-14 2004-02-26 Mitsubishi Chemical Corporation ANTIBODY SPECIFIC TO CENTRAL τ-PROTEIN
US8163896B1 (en) 2002-11-14 2012-04-24 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7906326B2 (en) 2003-05-07 2011-03-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides associated with alzheimer's disease and uses thereof
US20130130231A1 (en) 2002-11-26 2013-05-23 Isaac Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20060257851A1 (en) 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US7696334B1 (en) 2002-12-05 2010-04-13 Rosetta Genomics, Ltd. Bioinformatically detectable human herpesvirus 5 regulatory gene
US20070077553A1 (en) 2003-10-30 2007-04-05 Rosetta Genomics Bioinformatically detectable group of novel vaccinia regulatory genes and uses thereof
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
US7888497B2 (en) 2003-08-13 2011-02-15 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
AU2003904328A0 (en) 2003-08-13 2003-08-28 Garvan Institute Of Medical Research Diagnosis and treatment of neurodegenerative disorders
AU2004269150C1 (en) 2003-08-28 2010-11-18 Novartis Ag Interfering RNA duplex having blunt-ends and 3'-modifications
WO2005040382A2 (en) 2003-10-28 2005-05-06 Alexander Cherkasky Cherkasky fusion proteins containing antibody-, antigen- and microtubule-binding regions and immune response-triggering regions
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
CA2566519C (en) * 2004-05-14 2020-04-21 Rosetta Genomics Ltd. Micrornas and uses thereof
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
EP4047096A1 (en) 2004-06-28 2022-08-24 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7312035B2 (en) 2004-09-03 2007-12-25 Affymetrix, Inc. Methods of genetic analysis of yeast
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
US7833513B2 (en) 2004-12-03 2010-11-16 Rhode Island Hospital Treatment of Alzheimer's Disease
WO2006060753A2 (en) 2004-12-03 2006-06-08 Rhode Island Hospital Diagnosis and treatment of alzheimer's disease
US20070048756A1 (en) 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
EP1937312B1 (en) 2005-08-30 2016-06-29 Ionis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US7901882B2 (en) 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
CA3042781C (en) 2006-04-03 2021-10-19 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
US20090280110A1 (en) 2006-06-27 2009-11-12 University Of Virginia Patent Foundation Cell Model for Alzheimer's Disease Pathology
AU2007323469B2 (en) 2006-11-23 2014-07-24 Guangdong Maijinjia Biotechnology Co., Ltd Oligonucleotides for modulating target RNA activity
CA2682497C (en) 2007-04-05 2017-08-08 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
JP2008307007A (en) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag Human pluripotent stem cell induced from human tissue-originated undifferentiated stem cell after birth
WO2009109665A1 (en) 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
ES2599979T3 (en) 2009-04-24 2017-02-06 Roche Innovation Center Copenhagen A/S Pharmaceutical compositions for the treatment of HCV patients who do not respond to interferon
CN103003430A (en) 2009-11-12 2013-03-27 西澳大利亚大学 Antisense molecules and methods for treating pathologies
WO2011094645A1 (en) 2010-01-28 2011-08-04 University Of Massachusetts Lowell Methods of diagnosing tau-associated neurodegenerative diseases
WO2011117353A1 (en) 2010-03-24 2011-09-29 Mirrx Therapeutics A/S Bivalent antisense oligonucleotides
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
US9145557B2 (en) 2010-11-23 2015-09-29 Mirrx Therapeutics A/S Oligonucleotides for modulation of target RNA activity
EP2655621B1 (en) 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2012149438A1 (en) * 2011-04-28 2012-11-01 Life Technologies Corporation Methods and compositions for multiplex pcr
WO2013013165A2 (en) 2011-07-21 2013-01-24 The Trustees Of The University Of Pennsylvania Use of lna compounds for therapeutic inhibition of mir-21 in systemic lupus erythematosus
WO2013148260A1 (en) * 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
AU2013243946A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
JP6321633B2 (en) 2012-06-04 2018-05-09 ノバルティス アーゲー Site-specific labeling method and molecules produced thereby
WO2014064187A1 (en) 2012-10-24 2014-05-01 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. A microtubuli-modifying compound
US9200068B2 (en) 2012-12-18 2015-12-01 Regents Of The University Of Minnesota Compositions and methods related to tauopathy
KR20150099804A (en) 2012-12-20 2015-09-01 사렙타 쎄러퓨틱스 인코퍼레이티드 Improved exon skipping compositions for treating muscular dystrophy
EA202090946A3 (en) 2013-03-14 2021-07-30 Сарепта Терапьютикс, Инк. COMPOSITIONS PROVIDING EXONE LEAKING FOR THE TREATMENT OF MUSCLE DYSTROPHY
US9644207B2 (en) 2013-03-14 2017-05-09 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Tau expression
CA2903872A1 (en) 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
CA2951816A1 (en) 2013-06-12 2014-12-18 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
TW202246503A (en) * 2013-07-19 2022-12-01 美商百健Ma公司 Compositions for modulating tau expression
WO2015068075A2 (en) 2013-10-24 2015-05-14 Institut National De La Sante Et De La Recherche Medicale Detection of tau
WO2016011123A1 (en) * 2014-07-16 2016-01-21 Arrowhead Research Corporation Organic compositions to treat apoc3-related diseases
US20170211064A1 (en) 2014-07-29 2017-07-27 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
EP3254104B1 (en) 2015-02-04 2021-08-25 Bristol-Myers Squibb Company Methods of selecting therapeutic molecules
US20180023081A1 (en) 2015-02-04 2018-01-25 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
AU2016215155A1 (en) * 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
US20170035860A1 (en) * 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases

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